Formerly HCD Economics, Daresbury, UK.
HCD Economics, Daresbury, UK.
Eur J Health Econ. 2023 Feb;24(1):27-38. doi: 10.1007/s10198-022-01449-7. Epub 2022 Mar 26.
Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT.
Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews.
The mean age of participants was 41.50 years (SD 16.01, range 18-81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT.
This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies.
β-地中海贫血症（BT）是一种遗传性血液病，其特征是功能性血红蛋白水平降低，导致从临床无症状到严重贫血等多种表型。BT 患者可能需要终生定期输血，并辅以适当的铁螯合疗法（ICT）。本研究旨在确定英国普通人群如何看待与不同输血负担和 ICT 相关的 BT 健康状况。
采用复合时间权衡（cTTO）方法来评估 BT 相关健康状况的效用。在起草健康状况描述时，考虑了与症状和生活质量影响相关的 BT 文献，包括身体、功能和情感健康，以及 BT 治疗的安全性概况。制定了 11 种健康状况描述，并由血液学家和患者倡导者进行了临床准确性和完整性验证。英国普通人群中有 200 人参与了 cTTO 访谈。
参与者的平均年龄为 41.50 岁（标准差 16.01，范围 18-81）；88 人（46.8%）为女性。效用值范围从依赖输血的 BT 口服 ICT 的非输血依赖型的 0.78（标准差 0.34）到依赖输血的 BT 中高输血负担的皮下 ICT 的 0.37（标准差 0.50）。
本研究从英国普通人群的角度提供了一系列 BT 健康状况的健康效用。重要的是，较低的输血负担和较低的贫血负担与较高的效用相关。在较小程度上，ICT 的不同模式被发现对 BT 患者的效用评估有影响。本研究中获得的效用可作为 BT 治疗成本效益分析的投入。
